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    Muscle-specific expression vectors
    1.
    发明申请
    Muscle-specific expression vectors 审中-公开
    肌肉特异性表达载体

    公开(公告)号：US20110212529A1

    公开(公告)日：2011-09-01

    申请号：US11716899

    申请日：2007-03-12

    申请人： David Souza ,  Donna Armentano

    发明人： David Souza ,  Donna Armentano

    IPC分类号： C12N15/85 ,  C07H21/04 ,  C12N1/00 ,  C12N5/10

    CPC分类号： C12N15/85 ,  C12N2830/008 ,  C12N2830/85

    摘要： The invention is directed to novel combinations of muscle-specific enhancers and promoter elements useful for achieving persistent expression in the muscle or myocyctes. The muscle-specific promoter elements are derived from a muscle creatine kinase promoter, a troponin I promoter, a skeletal alpha-actin promoter, or a desmin promoter. The muscle-specific enhancer elements are derived from either troponin I internal regulatory elements, muscle creatine kinase enhancers, or desmin enhancers.

    摘要翻译： 本发明涉及用于在肌肉或肌细胞中实现持续表达的肌肉特异性增强子和启动子元件的新颖组合。 肌肉特异性启动子元件来源于肌肉肌酸激酶启动子，肌钙蛋白I启动子，骨骼α-肌动蛋白启动子或结蛋白启动子。 肌肉特异性增强子元件来自肌钙蛋白I内调节元件，肌肉肌酸激酶增强子或结蛋白增强子。

    Transgene expression systems
    2.
    发明授权
    Transgene expression systems 失效
    转基因表达系统

    公开(公告)号：US06358507B1

    公开(公告)日：2002-03-19

    申请号：US09416673

    申请日：1999-10-12

    申请人： Johanne Kaplan ,  Donna Armentano ,  Richard J. Gregory

    发明人： Johanne Kaplan ,  Donna Armentano ,  Richard J. Gregory

    IPC分类号： A61K4800

    CPC分类号： C12N15/86 ,  A61K38/1709 ,  A61K48/00 ,  C12N2710/10322 ,  C12N2710/10343

    摘要： The present invention relates to transgene expression systems, related compositions comprising the transgene expression systems, and methods of making and using them. Preferred systems employ an adenovirus transgene expression vector comprising DNA encoding a transgene which codes for a desired product operably linked to expression control sequence, and at least a portion of the adenovirus E3 region and certain portions of the E4 region. The E4 portions comprise the open reading frame sequence known as E40RF3 and at least one other portion of E4. Preferably the E4 portion of the vector (or “E4 cassette”) includes E40RF3 and at least one other portion selected from E40RF4, E40RF6/7 and E40RF3/4. The invention has a number of important features including improving persistency of transgene expression in a desired host cell. The transgene expression systems of the present invention are useful for a variety of applications including providing persistent cellular expression of the transgene in vitro and in vivo.

    摘要翻译： 本发明涉及转基因表达系统，包含转基因表达系统的相关组合物，以及制备和使用它们的方法。 优选的系统使用腺病毒转基因表达载体，其包含编码转基因的DNA，所述转基因编码与表达控制序列可操作地连接的所需产物，以及腺病毒E3区域的至少一部分和E4区域的某些部分。 E4部分包括被称为E40RF3的开放阅读框序列和E4的至少一个其他部分。 优选地，载体（或“E4盒”）的E4部分包括E40RF3和至少一个选自E40RF4，E40RF6 / 7和E40RF3 / 4的其它部分。 本发明具有许多重要特征，包括改善所需宿主细胞中转基因表达的持续性。 本发明的转基因表达系统可用于各种应用，包括在体外和体内提供转基因的持续细胞表达。

    Chimeric adenoviral vectors
    4.
    发明授权
    Chimeric adenoviral vectors 失效
    嵌合腺病毒载体

    公开(公告)号：US5877011A

    公开(公告)日：1999-03-02

    申请号：US752760

    申请日：1996-11-20

    申请人： Donna Armentano ,  Richard J. Gregory ,  Alan E. Smith

    发明人： Donna Armentano ,  Richard J. Gregory ,  Alan E. Smith

    IPC分类号： C12N15/09 ,  A61K48/00 ,  C07K14/075 ,  C12N15/861 ,  C12N15/86

    CPC分类号： C12N15/86 ,  C07K14/005 ,  C12N2710/10322 ,  C12N2710/10343 ,  C12N2810/6018

    摘要： A chimeric adenoviral vector is provided that comprises nucleotide sequence of a first adenovirus, wherein at least one gene of said first adenovirus encoding a protein that facilitates binding of said vector to a target mammalian cell, or internalization thereof within said cell, is replaced by the corresponding gene from a second adenovirus belonging to subgroup D, said vector further comprising a transgene operably linked to a eucaryotic promoter to allow for expression therefrom in a mammalian cell. Additionally, a method of delivering transgenes to target mammalian cells, particularly airway epithelial cells, is provided.

    摘要翻译： 提供了包含第一腺病毒的核苷酸序列的嵌合腺病毒载体，其中编码促进所述载体与靶哺乳动物细胞结合的蛋白质的所述第一腺病毒的至少一个基因或其在所述细胞内的内化被替换为 来自属于亚组D的第二腺病毒的相应基因，所述载体还包含可操作地连接到真核启动子以在哺乳动物细胞中从其中表达的转基因。 另外，提供了将转基因递送至靶哺乳动物细胞，特别是气道上皮细胞的方法。

    Regulatory elements for delivery to the liver
    7.
    发明授权
    Regulatory elements for delivery to the liver 有权
    用于输送到肝脏的调节元件

    公开(公告)号：US07312324B2

    公开(公告)日：2007-12-25

    申请号：US10139763

    申请日：2002-05-06

    申请人： David W. Souza ,  Donna Armentano ,  Samuel C. Wadsworth

    发明人： David W. Souza ,  Donna Armentano ,  Samuel C. Wadsworth

    IPC分类号： C07H21/04 ,  C07H21/02 ,  A01N63/00 ,  A01N65/00 ,  A01N43/04 ,  A01N31/70

    CPC分类号： C12N15/86 ,  A61K48/00 ,  A61K48/0058 ,  C12N15/85 ,  C12N2710/10343 ,  C12N2799/022 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/85

    摘要： The invention is directed to novel combinations of liver specific enhancers and promoter elements for achieving persistent transgene expression in the liver. The liver specific enhancer elements may be derived from either the human serum albumin, prothrombin, α-1microglobulin or aldolase genes in single copies or in multimerized form linked to elements derived from the cytomegalovirus intermediate early (CMV), α-1-antitrypsin or albumin promoters. In a preferred embodiment of the invention, an adenoviral vector comprising a liver specific enhancer/promoter combination operably linked to a transgene is administered to recipient cells. In other embodiments of the invention, adeno-associated viral vectors, retroviral vectors, lentiviral vectors or a plasmid comprising the liver specific enhancer/promoter combination linked to a transgene is administered to recipient cells. Also within the scope of the invention are promoter elements derived from the human prothrombin gene and the β-fibrinogen gene.

    摘要翻译： 本发明涉及用于实现肝脏中持续转基因表达的肝特异性增强子和启动子元件的新型组合。 肝脏特异性增强子元件可以来源于与来自巨细胞病毒中间体早期（CMV），α-1-抗胰蛋白酶或白蛋白的元件连接的单拷贝或多聚化形式的人血清白蛋白，凝血酶原，α-1微球蛋白或醛缩酶基因 发起人。 在本发明的优选实施方案中，向受体细胞施用包含可操作地连接到转基因的肝特异性增强子/启动子组合的腺病毒载体。 在本发明的其它实施方案中，将腺相关病毒载体，逆转录病毒载体，慢病毒载体或包含与转基因连接的肝特异性增强子/启动子组合的质粒施用于受体细胞。 也在本发明范围内的是源自人凝血酶原基因和β-纤维蛋白原基因的启动子元件。